A Multicenter, Open-label Extension Study to Evaluate the Safety, Pharmacodynamics, and Clinical Effects of WVE-120101 in Patients With Huntington's Disease
试验速览
- 阶段
- 1 期
- 状态
- 终止
- 入组人数
- 27
- 试验地点
- 18
- 主要终点
- Safety and Tolerability: Number of Patients Who Withdraw Due to TEAEs
研究概览
简要总结
WVE-HDSNP1-002 is an open-label extension (OLE) study to evaluate the safety, tolerability, PK, PD, and clinical effects of WVE-120101 in adult patients with early manifest HD who carry a targeted single nucleotide polymorphism, rs362307 (SNP1). To participate in the study, patients must have completed the Phase 1b/2a clinical study WVE-HDSNP1-001.
研究设计
- 研究类型
- Interventional
- 分配方式
- Na
- 干预模型
- Single Group
- 主要目的
- Treatment
- 盲法
- None
入排标准
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Patient successfully completed the Phase 1b/2a study with WVE-120101, WVE-HDSNP1-
排除标准
- •Received an investigational drug other than WVE-120101, including an investigational oligonucleotide, within the past 1 year or 5 half-lives of the drug, whichever is longer.
- •Inability to undergo brain MRI (with or without sedation).
- •Clinically significant medical finding on the physical examination other than HD that, in the judgment of the Investigator, will make the patient unsuitable for participation in and/or completion of the study procedures.
研究组 & 干预措施
WVE-120101 (Dose A)
干预措施: WVE-120101 (Drug)
结局指标
主要结局
Safety and Tolerability: Number of Patients Who Withdraw Due to TEAEs
时间窗: First dose received (Day 1) through the Study Termination visit (maximum of 45 weeks of treatment
Safety: Number of Patients With Treatment-emergent AEs (TEAEs)
时间窗: First dose received (Day 1) through the Study Termination visit (maximum of 45 weeks of treatment)
Safety: Number of Patients With Serious TEAEs
时间窗: First dose received (Day 1) through the Study Termination visit (maximum of 45 weeks of treatment
Safety: Number of Patients With a Severe TEAE
时间窗: First dose received (Day 1) through the Study Termination visit (maximum of 45 weeks of treatment
次要结局
未报告次要终点